FDA Expands Indications for Ilaris for Three Rare Diseases

Drug previously approved for cryopyrin-associated periodic syndromes and active systemic juvenile idiopathic arthritis

The FDA has approved three new indications for canakinumab (Ilaris, Novartis). The new indications are for rare and serious autoinflammatory diseases in adult and pediatric patients: tumor necrosis factor receptor-associated periodic syndrome (TRAPS); hyperimmunoglobulin D syndrome (HIDS)/mevalonate kinase deficiency (MKD); and familial Mediterranean fever.

All three syndromes are hereditary diseases that are characterized by periodic attacks of fever and inflammation, as well as severe muscle pain. There are no previously approved therapies for TRAPS or HIDS/MKD.

“For the first time, patients with TRAPS and HIDS/MKD, two painful and life-altering diseases, have access to a treatment that may help improve their quality of life,” said Badrul Chowdhury, MD, PhD, Director of the Division of Pulmonary, Allergy, and Rheumatology Products in FDA’s Center for Drug Evaluation and Research.

Canakinumab was previously approved for another periodic fever syndrome called cryopyrin-associated periodic syndromes and for active systemic juvenile idiopathic arthritis. Health care professionals should review the prescribing information in the labeling for detailed information about the approved uses.

Approvals for the new indications were based on clinical studies, including safety, efficacy, and pharmacokinetic data.

The most common adverse reactions for these indications are injection-site reactions and increased susceptibility to catching colds. Canakinumab can cause serious side effects, including increased risk of serious infections. Canakinumab can lower the immune system’s ability to fight infections.  The drug has also been linked with allergic reactions. Patients experiencing any symptoms of an allergic reaction (rash, itching and hives, difficulty breathing or swallowing, and dizziness or feeling faint) should call their health care provider. Patients should not get live vaccines if receiving canakinumab. Patients should not receive canakinumab if they are allergic to canakinumab or any of the ingredients in Ilaris.

Source: FDA; September 23, 2016.